CABA-201 for Inflammatory Myopathies

Recruiting at 39 trial locations

Overseen By Cabaletta Bio

Age: Any Age

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Cabaletta Bio

Disqualifiers: Severe infections, Organ transplant, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 1 day

24 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This clinical trial tests a new treatment called CABA-201 for individuals with certain muscle inflammation conditions, such as dermatomyositis (DM) and juvenile idiopathic inflammatory myopathy (JIIM). The trial aims to determine if CABA-201 is safe and effective in reducing symptoms like muscle weakness and skin rashes. Participants will receive an infusion of CABA-201 after preparation with other medications. Eligible individuals have been diagnosed with these conditions and continue to experience active symptoms despite treatment. As a Phase 1 trial, this research focuses on understanding how CABA-201 works in people, offering participants the opportunity to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.

Is there any evidence suggesting that CABA-201 is likely to be safe for humans?

Research has shown that CABA-201 is generally safe. In earlier studies, most patients did not experience serious side effects such as CRS (a reaction where the immune system releases too many proteins) or ICANS (a condition affecting the brain and nervous system). Only three out of eight patients had mild CRS, indicating that most people handled the treatment well. These results suggest that the treatment could be a good option for those considering joining the trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about CABA-201 for treating inflammatory myopathies because it offers a novel approach by targeting specific immune responses. Unlike standard treatments, which often involve broad immunosuppressants like corticosteroids or methotrexate, CABA-201 is infused following a tailored preconditioning regimen using fludarabine and cyclophosphamide. This combination aims to enhance the treatment's effectiveness by preparing the immune system to better respond to CABA-201. This targeted approach may lead to more precise control of the disease with potentially fewer side effects.

What evidence suggests that CABA-201 might be an effective treatment for inflammatory myopathies?

Research has shown that CABA-201 offers promising results for treating certain muscle inflammation diseases. In this trial, participants with dermatomyositis (DM), antisynthetase syndrome (ASyS), immune-mediated necrotizing myopathy (IMNM), and juvenile idiopathic inflammatory myopathies (JIIM) will receive an infusion of CABA-201 after preconditioning with fludarabine and cyclophosphamide. Previous studies with patients who have DM and ASyS indicated that CABA-201 reduced symptoms by lowering immune system activity, which is crucial in these conditions. Additionally, early results from individuals with IMNM showed significant improvements. While more research is needed, these initial findings suggest that CABA-201 could effectively treat these inflammatory muscle diseases. ^{1 2 4 5 6}

Who Is on the Research Team?

Medical Director

Principal Investigator

Cabaletta Bio

Are You a Good Fit for This Trial?

This trial is for adults aged 18-65 with active idiopathic inflammatory myopathy (IIM), including conditions like dermatomyositis and antisynthetase syndrome. Participants must have evidence of ongoing disease despite treatment, shown by muscle weakness or tests like MRI. Excluded are those with severe reactions to the study drugs, active infections needing treatment, major organ issues, previous CAR T cell therapy, or any transplants.

Inclusion Criteria

I experience muscle weakness.

My condition is active despite treatment, shown by tests like MRI or high CK levels.

I have been diagnosed with a muscle condition due to specific antibodies in my blood.

Exclusion Criteria

I am currently being treated for an infection.

I cannot undergo leukapheresis due to health reasons.

I have received CAR T cell therapy before.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Preconditioning

Participants receive preconditioning with fludarabine and cyclophosphamide

1 week

Treatment

Participants receive a single infusion of CABA-201

1 day

Follow-up

Participants are monitored for safety and effectiveness after treatment

156 weeks

What Are the Treatments Tested in This Trial?

Interventions

• CABA-201

Trial Overview The trial is testing CABA-201 after patients receive fludarabine and cyclophosphamide preconditioning. It's an open-label study which means everyone knows they're getting the experimental treatment and there's no placebo group.

How Is the Trial Designed?

Group I: CABA-201 Experimental Treatment 1 Intervention

DM Cohort: Infusion of CABA-201 following preconditioning with fludarabine and cyclophosphamide preconditioning in subjects with DM. ASyS Cohort: Infusion of CABA-201 following preconditioning with fludarabine and cyclophosphamide preconditioning in subjects with ASyS. IMNM Cohort: Infusion of CABA-201 following preconditioning with fludarabine and cyclophosphamide preconditioning in subjects with IMNM. JIIM Cohort: Infusion of CABA-201 following preconditioning with fludarabine and cyclophosphamide preconditioning in subjects with JIIM.

CABA-201 is already approved in United States for the following indications:

Approved in United States as CABA-201 for:

• None approved; under investigation for systemic lupus erythematosus, myositis, systemic sclerosis, myasthenia gravis, and pemphigus vulgaris

Who Is Running the Clinical Trial?

Cabaletta Bio

Lead Sponsor

Trials

6

Recruited

140+

Citations

1. cabalettabio.com cabalettabio.com/patients/phase-12-trial-in-myositis

RESET-Myositis™ Phase 1/2 Trial RESET-Myositis is a phase 1/2 clinical trial being conducted to evaluate the effects of the investigational cell treatment CABA-201 in patients with ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06154252

NCT06154252 | RESET-Myositis: An Open-Label Study to ... This study is being conducted to evaluate the safety and efficacy of an investigational cell therapy, CABA-201, that can be given to patients with DM, ASyS, ...

3. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39245937/

Case study of CD19 CAR T therapy in a subject with ... These data detail the safety, efficacy, and pharmacodynamics of CABA-201 in the first IMNM subject. Copyright © 2024 Cabaletta Bio. Published by ...

4. cabalettabio.com cabalettabio.com/news-media/press-releases/detail/137/cabaletta-bio-presents-positive-clinical-data-and

Cabaletta Bio Presents Positive Clinical Data and ... Cabaletta is presenting complete adult Phase 1/2 clinical data from 6 patients in the combined DM/ASyS (4 dermatomyositis and 2 antisynthetase ...

5. acrabstracts.org acrabstracts.org/abstract/safety-and-efficacy-of-caba-201-a-fully-human-autologous-4-1bb-anti-cd19-car-t-cell-therapy-in-patients-with-immune-mediated-necrotizing-myopathy-and-systemic-lupus-erythematosus-from-the-reset-myos/

Safety and Efficacy of CABA-201, a Fully Human ... Results: Baseline data on the 2 treated subjects are in Table 1. In both subjects, CABA-201 expansion peaked at day 15 and peripheral B cells were rapidly ...

6. cabalettabio.com cabalettabio.com/investors/news-events/press-releases/detail/119/cabaletta-bio-presents-positive-clinical-safety-and

Cabaletta Bio Presents Positive Clinical Safety and Efficacy ... CABA-201 safety profile suggests favorable risk-benefit with no CRS or ICANS in the majority of patients; low-grade CRS in three of eight ...